Amyloidosis
Conditions
Brief summary
The purpose of the study is to evaluate the safety and tolerability of single dose of NPT189 in healthy volunteers. The study will also evaluate the pharmacokinetic characteristics of NPT189.
Detailed description
This study is a Phase1, randomized, double-blind, placebo-controlled, single dose, dose escalation safety and tolerability study of NPT189 in health subjects. Six dose cohorts are planned each with a maximum of 8 participants per cohort. Participants will receive NPT189 by intravenous (IV) or a matching placebo by intravenous infusion. Safety, tolerability, and pharmacokinetics will be assessed.
Interventions
DRUGNPT189
IgG1 Fc-GAIM fusion protein, a recombinant fusion protein
DRUGPlacebo
Placebo
Sponsors
Proclara Biosciences, Inc.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to 65 Years
Healthy volunteers
Yes
Inclusion criteria
* Male or female subjects between the ages of 18 and 65 years (inclusive); * Has provided written informed consent to participate in the study; * Weigh ≥ 45kg and ≤ 120kg with Body Mass Index of 18-32 inclusive; * Subject Rescreening: This study does not permit the re-enrollment of a subject who has discontinued from the study as a pre-treatment failure without approval from the sponsor unless the reason they were not included was because the required number of subjects needed at the time of randomization had already been met. Rescreened subjects must continue to meet all inclusion and
Exclusion criteria
. All screening procedures must be performed within 28 days of dose administration * Female subjects of childbearing potential (defined as not surgically sterile or at least two years postmenopausal confirmed by a Screening Follicle Stimulating Hormone (FSH) level of \> 40) must agree to use one of the following forms of contraception from 60 days prior through 90 days following the dose of Study Drug: hormonal (oral, transdermal, implant, or injection), barrier (condom, diaphragm with spermicide), intrauterine device (IUD), complete abstinence, or vasectomized partner (6 months minimum) or male subjects who are sexually active with women of child-bearing potential must agree to complete abstinence or to use a condom for 90 days following the dose of Study Drug; * No clinically significant abnormal findings on physical examination, vital signs, ECG, or clinical laboratory evaluation during screening; and systolic blood pressure between 140 mm and 90 mm Hg and diastolic blood pressure between 90 mm and 45 mm Hg. * Can understand and sign the informed consent document, can communicate with the investigator, and can understand and comply with the requirements of the protocol.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Safety of a single intravenous (IV) infusion of NPT189 (subjects experiencing adverse events) | Day 1 through to Day 49 | Summary of the frequency and percentage of |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Profile of pharmacokinetics (PK) of a single intravenous (IV) infusion of NPT189 (Tmax) | Day 1 through to Day 49 | Time of the maximum measured concentration (Tmax) |
| Profile of pharmacokinetics (PK) of a single intravenous (IV) infusion of NPT189 (AUClast) | Day 1 through to Day 49 | Area under the concentration-time curve from time zero to the last quantifiable concentration time-point (AUClast) |
| Profile of pharmacokinetics (PK) of a single intravenous (IV) infusion of NPT189 (AUCinf) | Day 1 through to Day 49 | Area under the concentration-time curve from time zero extrapolated to infinity (AUCinf) |
| Profile of pharmacokinetics (PK) of a single intravenous (IV) infusion of NPT189 (Cmax) | Day 1 through to Day 49 | Observed maximum concentration (Cmax) |
| Profile of pharmacokinetics (PK) of a single intravenous (IV) infusion of NPT189. Clearance (CL) | Day 1 through to Day 49 | Clearance (CL) |
| Profile of pharmacokinetics (PK) of a single intravenous (IV) infusion of NPT189. Volume of distribution (Vd) | Day 1 through to Day 49 | Apparent volume of distribution (Vd) |
| Profile of pharmacokinetics (PK) of a single intravenous (IV) infusion of NPT189. Terminal elimination half-life (t 1/2) | Day 1 through to Day 49 | Terminal elimination half-life (t 1/2) |
Countries
Netherlands
Outcome results
None listed